Hyperphagia in Prader-Willi Syndrome
Conditions
Keywords
Opel-label extension, safety, tolerability
Brief summary
To evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with PWS
Detailed description
This is a long-term, OLE study to evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with PWS. Subjects who complete the antecedent double-blind study (ACP-101-302) will be invited to participate in the present study.
Interventions
DRUGCarbetocin
Carbetocin nasal spray 3.2 mg three times daily (TID)
Sponsors
ACADIA Pharmaceuticals Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
5 Years to 30 Years
Healthy volunteers
No
Inclusion criteria
* Has completed the Week12/EOT visit of the antecedent, Study ACP-101-302 * Met all entry criteria for the antecedent study * May benefit from long-term treatment with open-label carbetocin in the judgment of the Investigator. * Lives with a caregiver who understands and is willing and able to adhere to study-related procedures and is willing to participate in all study visits.
Exclusion criteria
* History of, or current, cerebrovascular disease, brain trauma, epilepsy, or frequent migraines. A history of febrile seizures is not exclusionary. * Active psychotic symptoms, a history of psychotic symptoms, or a psychotic disorder * History of suicide attempt or inpatient psychiatric hospitalization * Has a clinically significant abnormality in vital signs at Baseline * Has an average QTcF interval of \>450 ms on the Baseline ECG performed before the first dose of carbetocin is given in the present study (i.e., the ECG performed at the EOT visit of the antecedent study) * Has developed a clinically significant ECG finding during the antecedent study * Subject is judged by the Investigator or the Medical Monitor to be inappropriate for the study due to AEs, medical condition, or noncompliance with investigational product or study procedures in the antecedent study Additional inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), withdrawals due to adverse events (AEs), potentially clinically important changes in other safety assessments, device incidents or device malfunctions | Baseline to 36 months | Treatment-emergent adverse events (TEAEs) and device incidents or device malfunctions, TEAEs leading to discontinuation, TEAEs related to study drug, TEAEs by maximum severity, fatal TEAEs, treatment-emergent SAEs, and treatment-emergent SAEs related to study drug will all be summarized for all subjects as well as by previous treatment group. |
Countries
Canada, France, Germany, Spain, United Kingdom, United States
Outcome results
None listed